A Study Evaluating the Safety and Efficacy of EDIT-301 in Participants With Severe Sickle Cell Disease (RUBY) (NCT04853576)
A Phase 1/2 Study to Evaluate the Safety and Efficacy of a Single Dose of Autologous Clustered Regularly Interspaced Short Palindromic Repeats Gene-edited CD34+ Human Hematopoietic Stem and Progenitor Cells (EDIT-301) in Subjects With Severe Sickle Cell Disease
๐ฉบ Plain-English Summary (8th-Grade Level)
The purpose of this study is to evaluate the efficacy, safety and tolerability of treatment with EDIT-301 in adult and adolescent participants with severe sickle cell disease (SCD).
Who can join: Review the eligibility criteria below.
Location: UCSF Benioff Children's Hospital โ Oakland, California
Age range: 12 Years to 50 Years
๐งช Interventions in This Study
- EDIT-301 (GENETIC)
๐ "Do I Qualify?" โ 1-Minute Self Screener
Instant ChecklistFull eligibility criteria are available on the official NIH protocol. View the criteria โ
๐ All Participating Trial Locations (24 Sites)
- UCSF Benioff Children's Hospital โ Oakland, California
- Children's Hospital Colorado โ Aurora, Colorado
- Smilow Cancer Hospital โ New Haven, Connecticut
- Johns Hopkins All Children's Hospital โ St. Petersburg, Florida
- Children's Healthcare of Atlanta โ Atlanta, Georgia
- Ann & Robert H. Lurie Children's Hospital of Chicago โ Chicago, Illinois
- University of Mississippi Medical Center โ Jackson, Mississippi
- Hackensack University Medical Center โ Hackensack, New Jersey
- Columbia University Medical Center - Department of Pediatrics โ New York, New York
- Columbia University Medical Center โ New York, New York
- The University of North Carolina at Chapel Hill โ Chapel Hill, North Carolina
- Atrium Health โ Charlotte, North Carolina
- University Hospitals Rainbow Babies & Children's Hospital โ Cleveland, Ohio
- Cleveland Clinic โ Cleveland, Ohio
- Nationwide Children's Hospital โ Columbus, Ohio
- The James Cancer Hospital โ Columbus, Ohio
- Children's Hospital of Philadelphia โ Philadelphia, Pennsylvania
- Medical University of South Carolina โ Charleston, South Carolina
- Tristar Medical Group Children's Specialists/Sarah Cannon Center for Blood Cancers โ Nashville, Tennessee
- Texas Oncology - Baylor Charles A. Sammons Cancer Center โ Dallas, Texas
- Cook Children's โ Fort Worth, Texas
- Ottawa Hospital Research Institute โ Ottawa, Ontario
- Princess Margaret Cancer Centre โ Toronto, Ontario
- Centre Hospitalier Universitaire Sainte-Justine โ Montreal, Quebec
๐ Contact the Study Coordinator
Phone: (877) 827-3222
Coordinator details come directly from the registered NIH protocol. Confirm the study is still recruiting before traveling.
๐ฐ Cost, Insurance & Patient Rights
- 100% Free Items: The investigational treatment and protocol-required procedures are supplied at zero cost by the sponsor.
- Routine Patient Care: Under federal law (ACA ยง 2709) and many state statutes, routine care costs are covered during participation.
- Voluntary Participation: You may withdraw at any time without affecting your standard medical care.
More studies in Sickle Cell Disease
- Kidney Function in Sickle Cell Anemia โ Chapel Hill, North Carolina
- BEACON: A Study Evaluating the Safety and Efficacy of BEAM-101 in Patients With Severe Sickle Cell Disease โ Birmingham, Alabama
- Nonmyeloablative Haploidentical Peripheral Blood Mobilized Hematopoietic Precursor Cell Transplantation for Sickle Cell Disease โ Bethesda, Maryland
- Acupressure in Patients With Sickle Cell Disease โ Irvine, California
- Long-term Follow-up (LTFU) of Patients Treated With Genome-edited Autologous Hematopoietic Stem and Progenitor Cells (HSPC) โ Chicago, Illinois
- Evaluation of Safety and Efficacy of CTX001 in Pediatric Participants With Severe Sickle Cell Disease (SCD) โ Charlotte, North Carolina
Explore by condition: All Sickle Cell Disease clinical trials
๐ก How to Participate
- Check the checklist above to see if you may qualify.
- Contact the study coordinator using the phone/email above.
- Ask about the visit schedule, what is covered at no cost, and any travel support.
- Confirm with your own doctor before making a decision.